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Researchers develop 'dimmer switch' to help control gene therapy

In a major advancement in the field of gene therapy for rare and devastating diseases, researchers have developed a "dimmer switch" system that can control levels of proteins expressed from gene therapy vectors. The system is based on alternative RNA splicing using an orally available small molecule and works effectively in tissues throughout the body, including the brain.

source https://www.sciencedaily.com/releases/2021/07/210728111307.htm

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